La Jolla Pharmaceutical Company Announces Launch of Expanded Access
Program for LJPC-501

August 08, 2017 09:00 AM Eastern Daylight Time

SAN DIEGO--(BUSINESS WIRE)--La
Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La
Jolla), today announced that the Company has initiated an expanded
access program (EAP) in the United States to provide its investigational
drug, LJPC-501 (angiotensin II), to patients with vasodilatory or
distributive shock who cannot achieve target mean arterial pressure
(MAP) despite adequate fluid resuscitation and treatment with currently
available vasopressors (a syndrome alternatively known as “catecholamine
resistant hypotension” or “clinically refractory hypotension” (CRH)).

Expanded access, sometimes known as compassionate use, is an option
facilitated by the U.S. Food and Drug Administration (FDA) to make
available prior to regulatory approval investigational medicine(s) for
the treatment of serious or life-threatening diseases or conditions
where there are no ongoing clinical trials and there is a lack of
satisfactory therapeutic alternatives.

“We are pleased to commence this expanded access program for eligible
patients in the U.S.,” said George F. Tidmarsh, M.D., Ph.D., President
and Chief Executive Officer of La Jolla. “This EAP provides a mechanism
for eligible patients to access LJPC-501 while the Company is diligently
working to pursue FDA approval."

About LJPC-501 Expanded Access Program

The
LJPC-501 EAP is a program for U.S. patients 18 years of age or older
with vasodilatory or distributive shock who are unable to achieve target
MAP despite adequate fluid resuscitation and treatment with currently
available vasopressors. The enrollment criteria are available at www.clinicaltrials.gov.

Expanded access, sometimes called "compassionate use," is the use of an
investigational medical product to treat a patient’s serious or
life-threatening disease or condition outside of a clinical trial. The
main distinction between expanded access and the use of an
investigational medicine in clinical studies is that expanded access
programs are not intended to obtain information about the safety or
effectiveness of the drug. Expanded access to an investigational
medicinal product can only be provided if the sponsor is actively
pursuing marketing approval of the drug. The investigational medicine(s)
made available through expanded access have not yet received regulatory
approval; therefore, their potential safety and efficacy have not yet
been established by the FDA. Doctors and patients should consider all
possible benefits and risks when seeking access to investigational
medicine(s) prior to regulatory approval.

Under 21 CFR 312.305(a)(3), to authorize any category of expanded
access, FDA must determine that expanded access to the drug for the
requested use will not interfere with the initiation, conduct, or
completion of clinical investigations that could support marketing
approval of the expanded access use or otherwise compromise the
potential development of the drug for the expanded access use. In
addition, FDA must determine that there is sufficient evidence of the
safety and effectiveness of the investigational product to support its
use in the particular circumstance.

Once a regulatory agency approves the investigational medicine for
commercial use and the medicine is commercially available, existing
expanded access programs for that medicine will be phased out as soon as
practicable while not interfering with patient safety.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human
angiotensin II. Angiotensin II, the major bioactive component of the
renin-angiotensin system, serves as one of the body’s central regulators
of blood pressure. LJPC-501 is being developed for the treatment of
patients with catecholamine resistant hypotension (CRH). LJPC-501 is the
first synthetic human angiotensin II product candidate to be tested in a
Phase 3 study. In February 2017, La Jolla reported positive top-line
results from the ATHOS-3 (Angiotensin II for the Treatment
of High-Output Shock) Phase 3 study of LJPC-501 in patients with CRH.
In May 2017, the results of ATHOS-3 were published by The New England
Journal of Medicine in an article entitled “Angiotensin II for the
Treatment of Vasodilatory Shock.” The NEJM article and its Supplementary
Appendix can be found here.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension or clinically refractory hypotension
(CRH) is a life-threatening syndrome in patients with vasodilatory (also
known as distributive) shock (dangerously low blood pressure with
adequate cardiac function) who cannot achieve target mean arterial
pressure (MAP) despite adequate fluid resuscitation and treatment with
currently available vasopressors (catecholamines and/or vasopressin).
There are approximately 500,000 distributive shock cases in the United
States per year, an estimated 200,000 of which develop CRH. On current
therapies, more than 50% of CRH patients die within 30 days.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused
on the discovery, development and commercialization of innovative
therapies intended to significantly improve outcomes in patients
suffering from life-threatening diseases. The Company has several
product candidates in development. LJPC-501 is La Jolla’s proprietary
formulation of synthetic human angiotensin II for the potential
treatment of catecholamine resistant hypotension. LJPC-401 is La Jolla’s
proprietary formulation of synthetic human hepcidin for the potential
treatment of conditions characterized by iron overload, such as
hereditary hemochromatosis, beta thalassemia, sickle cell disease and
myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation
gentamicin derivative program that is focused on the potential treatment
of serious bacterial infections as well as rare genetic disorders, such
as cystic fibrosis and Duchenne muscular dystrophy. For more information
on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is
defined in the Private Securities Litigation Reform Act of 1995. These
statements relate to future events or the Company’s future results of
operations. These statements are only predictions or statements of
current expectations and involve known and unknown risks, uncertainties
and other factors, that may cause actual results to be materially
different from those anticipated by the forward-looking statements. The
Company cautions readers not to place undue reliance on any such
forward-looking statements, which speak only as of the date they were
made. Certain of these risks, uncertainties, and other factors are
described in greater detail in the Company’s filings with the U.S.
Securities and Exchange Commission (SEC), all of which are available
free of charge on the SEC’s web site www.sec.gov.
These risks include, but are not limited to, risks relating to: the
timing and availability of LJPC-501 under the expanded access program;
the timing and prospects for approval of LJPC-501 by the FDA and the
other regulatory authorities; risks relating to the scope of product
labels (if approved) and potential market sizes, as well as the broader
commercial opportunity; the anticipated timing for regulatory actions;
and the success of future development activities; potential indications
for which the Company’s product candidates may be developed. The Company
expressly disclaims any intent to update any forward-looking statements
to reflect the outcome of subsequent events.